Urea cycle disorders - Pipeline Insight, 2021
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DelveInsight’s, “Urea cycle disorders - Pipeline Insight, 2021,” report provides comprehensive insights about 8+ companies and 8+ pipeline drugs in Urea cycle disorders pipeline landscape. It covers the pipeline drug profiles, including clinical and nonclinical stage products. It also covers the therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.
Geography Covered
Urea cycle disorders: Overview
A urea cycle disorder (UCD) is an inherited disease. When we eat protein our bodies break it down into amino acids which we need to grow and stay healthy. Extra amino acids that our body doesn’t need are turned into a waste product called ammonia. Enzymes in the liver turn the ammonia into urea so the body can get rid of it through the urine. In someone with a UCD, the enzyme is missing or is not working correctly. Because of this, ammonia builds up in the blood and it can be harmful. It can travel to the brain and cause memory loss, behavior changes, coma, seizures, or brain damage. UCDs affect about 1 in 35,000 births in the United States. Just because it’s rare doesn’t mean there isn’t information to help you or someone you love who is living with a UCD.
'Urea cycle disorders - Pipeline Insight, 2021' report by DelveInsight outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Urea cycle disorders pipeline landscape is provided which includes the disease overview and Urea cycle disorders treatment guidelines. The assessment part of the report embraces, in depth Urea cycle disorders commercial assessment and clinical assessment of the pipeline products under development. In the report, detailed description of the drug is given which includes mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, Urea cycle disorders collaborations, licensing, mergers and acquisition, funding, designations and other product related details.
Report Highlights
This segment of the Urea cycle disorders report encloses its detailed analysis of various drugs in different stages of clinical development, including phase II, I, preclinical and Discovery. It also helps to understand clinical trial details, expressive pharmacological action, agreements and collaborations, and the latest news and press releases.
Urea cycle disorders Emerging Drugs
Further product details are provided in the report..
Urea cycle disorders: Therapeutic Assessment
This segment of the report provides insights about the different Urea cycle disorders drugs segregated based on following parameters that define the scope of the report, such as:
Urea cycle disorders: Pipeline Development Activities
The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Urea cycle disorders therapeutic drugs key players involved in developing key drugs.
Pipeline Development Activities
The report covers the detailed information of collaborations, acquisition and merger, licensing along with a thorough therapeutic assessment of emerging Urea cycle disorders drugs.
Urea cycle disorders Report Insights
Current Treatment Scenario and Emerging Therapies:
DelveInsight’s, “Urea cycle disorders - Pipeline Insight, 2021,” report provides comprehensive insights about 8+ companies and 8+ pipeline drugs in Urea cycle disorders pipeline landscape. It covers the pipeline drug profiles, including clinical and nonclinical stage products. It also covers the therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.
Geography Covered
- Global coverage
Urea cycle disorders: Overview
A urea cycle disorder (UCD) is an inherited disease. When we eat protein our bodies break it down into amino acids which we need to grow and stay healthy. Extra amino acids that our body doesn’t need are turned into a waste product called ammonia. Enzymes in the liver turn the ammonia into urea so the body can get rid of it through the urine. In someone with a UCD, the enzyme is missing or is not working correctly. Because of this, ammonia builds up in the blood and it can be harmful. It can travel to the brain and cause memory loss, behavior changes, coma, seizures, or brain damage. UCDs affect about 1 in 35,000 births in the United States. Just because it’s rare doesn’t mean there isn’t information to help you or someone you love who is living with a UCD.
'Urea cycle disorders - Pipeline Insight, 2021' report by DelveInsight outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Urea cycle disorders pipeline landscape is provided which includes the disease overview and Urea cycle disorders treatment guidelines. The assessment part of the report embraces, in depth Urea cycle disorders commercial assessment and clinical assessment of the pipeline products under development. In the report, detailed description of the drug is given which includes mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, Urea cycle disorders collaborations, licensing, mergers and acquisition, funding, designations and other product related details.
Report Highlights
- The companies and academics are working to assess challenges and seek opportunities that could influence Urea cycle disorders R&D. The therapies under development are focused on novel approaches to treat/improve Urea cycle disorders.
This segment of the Urea cycle disorders report encloses its detailed analysis of various drugs in different stages of clinical development, including phase II, I, preclinical and Discovery. It also helps to understand clinical trial details, expressive pharmacological action, agreements and collaborations, and the latest news and press releases.
Urea cycle disorders Emerging Drugs
- ACER-001: Acer Therapeutics
- Avalotcagene ontaparvovec: Dimension Therapeutics
Further product details are provided in the report..
Urea cycle disorders: Therapeutic Assessment
This segment of the report provides insights about the different Urea cycle disorders drugs segregated based on following parameters that define the scope of the report, such as:
- Major Players in Urea cycle disorders
- Phases
- Late stage products (Phase III)
- Mid-stage products (Phase II)
- Early-stage product (Phase I) along with the details of
- Pre-clinical and Discovery stage candidates
- Discontinued & Inactive candidates
- Route of Administration
- Oral
- Parenteral
- intravitreal
- Subretinal
- Topical.
- Molecule Type
- Monoclonal Antibody
- Peptides
- Polymer
- Small molecule
- Gene therapy
- Product Type
Urea cycle disorders: Pipeline Development Activities
The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Urea cycle disorders therapeutic drugs key players involved in developing key drugs.
Pipeline Development Activities
The report covers the detailed information of collaborations, acquisition and merger, licensing along with a thorough therapeutic assessment of emerging Urea cycle disorders drugs.
Urea cycle disorders Report Insights
- Urea cycle disorders Pipeline Analysis
- Therapeutic Assessment
- Unmet Needs
- Impact of Drugs
- Pipeline Product Profiles
- Therapeutic Assessment
- Pipeline Assessment
- Inactive drugs assessment
- Unmet Needs
Current Treatment Scenario and Emerging Therapies:
- How many companies are developing Urea cycle disorders drugs?
- How many Urea cycle disorders drugs are developed by each company?
- How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Urea cycle disorders?
- What are the key collaborations (Industry–Industry, Industry–Academia), Mergers and acquisitions, licensing activities related to the Urea cycle disorders therapeutics?
- What are the recent trends, drug types and novel technologies developed to overcome the limitation of existing therapies?
- What are the clinical studies going on for Urea cycle disorders and their status?
- What are the key designations that have been granted to the emerging drugs?
- Acer Therapeutics
- Dimension Therapeutics
- Callitas Therapeutics
- Poseida Therapeutics
- Promethera Biosciences
- Arcturus Therapeutics
- Kaleido Biosciences
- Akaza Biopharma
- Evox Therapeutics
- Dipharma SA
- Sana Biotechnology
- Aeglea BioTherapeutics
- ERYTECH
- ACER-001
- Avalotcagene ontaparvovec
- MACS
- P OTC 101
- Heparesc
- ARCT 810
- VS 01
- KB195
- HepaStem
- Resatorvid
- EVX-103
- SG 328
- Pegzilarginase
- ERY-ADI
Introduction
Executive Summary
Urea cycle disorders: Overview
Causes
Mechanism of Action
Signs and Symptoms
Diagnosis
Disease Management
Pipeline Therapeutics
Comparative Analysis
Therapeutic Assessment
Assessment by Product Type
Assessment by Stage and Product Type
Assessment by Route of Administration
Assessment by Stage and Route of Administration
Assessment by Molecule Type
Assessment by Stage and Molecule Type
Urea cycle disorders – DelveInsight’s Analytical Perspective
In-depth Commercial Assessment
Urea cycle disorders companies’ collaborations, Licensing, Acquisition -Deal Value Trends
Urea cycle disorders Collaboration Deals
Company-Company Collaborations (Licensing / Partnering) Analysis
Company-University Collaborations (Licensing / Partnering) Analysis
Late Stage Products (Phase III)
Comparative Analysis
ACER-001: Acer Therapeutics
Product Description
Research and Development
Product Development Activities
Drug profiles in the detailed report..
Mid Stage Products (Phase II)
Comparative Analysis
Avalotcagene ontaparvovec: Dimension Therapeutics
Product Description
Research and Development
Product Development Activities
Drug profiles in the detailed report..
Inactive Products
Comparative Analysis
Urea cycle disorders Key Companies
Urea cycle disorders Key Products
Urea cycle disorders- Unmet Needs
Urea cycle disorders- Market Drivers and Barriers
Urea cycle disorders- Future Perspectives and Conclusion
Urea cycle disorders Analyst Views
Urea cycle disorders Key Companies
Appendix
Executive Summary
Urea cycle disorders: Overview
Causes
Mechanism of Action
Signs and Symptoms
Diagnosis
Disease Management
Pipeline Therapeutics
Comparative Analysis
Therapeutic Assessment
Assessment by Product Type
Assessment by Stage and Product Type
Assessment by Route of Administration
Assessment by Stage and Route of Administration
Assessment by Molecule Type
Assessment by Stage and Molecule Type
Urea cycle disorders – DelveInsight’s Analytical Perspective
In-depth Commercial Assessment
Urea cycle disorders companies’ collaborations, Licensing, Acquisition -Deal Value Trends
Urea cycle disorders Collaboration Deals
Company-Company Collaborations (Licensing / Partnering) Analysis
Company-University Collaborations (Licensing / Partnering) Analysis
Late Stage Products (Phase III)
Comparative Analysis
ACER-001: Acer Therapeutics
Product Description
Research and Development
Product Development Activities
Drug profiles in the detailed report..
Mid Stage Products (Phase II)
Comparative Analysis
Avalotcagene ontaparvovec: Dimension Therapeutics
Product Description
Research and Development
Product Development Activities
Drug profiles in the detailed report..
Inactive Products
Comparative Analysis
Urea cycle disorders Key Companies
Urea cycle disorders Key Products
Urea cycle disorders- Unmet Needs
Urea cycle disorders- Market Drivers and Barriers
Urea cycle disorders- Future Perspectives and Conclusion
Urea cycle disorders Analyst Views
Urea cycle disorders Key Companies
Appendix
LIST OF TABLES
Table 1 Total Products for Urea cycle disorders
Table 2 Late Stage Products
Table 3 Mid Stage Products
Table 4 Early Stage Products
Table 5 Pre-clinical & Discovery Stage Products
Table 6 Assessment by Product Type
Table 7 Assessment by Stage and Product Type
Table 8 Assessment by Route of Administration
Table 9 Assessment by Stage and Route of Administration
Table 10 Assessment by Molecule Type
Table 11 Assessment by Stage and Molecule Type
Table 12 Inactive Products
Table 1 Total Products for Urea cycle disorders
Table 2 Late Stage Products
Table 3 Mid Stage Products
Table 4 Early Stage Products
Table 5 Pre-clinical & Discovery Stage Products
Table 6 Assessment by Product Type
Table 7 Assessment by Stage and Product Type
Table 8 Assessment by Route of Administration
Table 9 Assessment by Stage and Route of Administration
Table 10 Assessment by Molecule Type
Table 11 Assessment by Stage and Molecule Type
Table 12 Inactive Products
LIST OF FIGURES
Figure 1 Total Products for Urea cycle disorders
Figure 2 Late Stage Products
Figure 3 Mid Stage Products
Figure 4 Early Stage Products
Figure 5 Preclinical and Discovery Stage Products
Figure 6 Assessment by Product Type
Figure 7 Assessment by Stage and Product Type
Figure 8 Assessment by Route of Administration
Figure 9 Assessment by Stage and Route of Administration
Figure 10 Assessment by Molecule Type
Figure 11 Assessment by Stage and Molecule Type
Figure 12 Inactive Products
Figure 1 Total Products for Urea cycle disorders
Figure 2 Late Stage Products
Figure 3 Mid Stage Products
Figure 4 Early Stage Products
Figure 5 Preclinical and Discovery Stage Products
Figure 6 Assessment by Product Type
Figure 7 Assessment by Stage and Product Type
Figure 8 Assessment by Route of Administration
Figure 9 Assessment by Stage and Route of Administration
Figure 10 Assessment by Molecule Type
Figure 11 Assessment by Stage and Molecule Type
Figure 12 Inactive Products
